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# FDA Approves Expanded Use for Vertex Kalydeco
- URL: https://www.fdaweb.com/fda-approves-expanded-use-for-vertex-kalydeco/
- Published: 2018-08-15T12:00:00.000Z
- Updated: 2026-09-15T00:25:57.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5142118

FDA has approved Vertex Pharmaceuticals’ Kalydeco (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to 24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to therapy based on clinical or *in vitro* assay data. Approval is based on data from an ongoing Phase 3 open-label safety study (ARRIVAL) of 25 children with CF who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). “The study demonstrated a safety profile consistent with that observed in previous Phase 3 studies of older children and adults; most adverse events were mild or moderate in severity, and no patient discontinued due to adverse events,” the company says. “Two patients had elevated liver enzymes greater than eight times the upper limit of normal, but continued to receive Kalydeco after a dose interruption,” it says.